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A gentle chemotherapy that switches silenced genes back on. With venetoclax it became the standard for older people with AML who cannot take intensive treatment.
Approved for MDS (2004) and, in combination with venetoclax, for newly diagnosed AML in patients unfit for intensive chemotherapy (VIALE-A, 2020). Oral azacitidine (Onureg, CC-486) is approved as maintenance after intensive induction (QUAZAR AML-001: OS 24.7 vs 14.8 months). Backbone partner for IDH inhibitors (AGILE), menin inhibitors, and FLT3 inhibitors in trials.
Cytidine analogue incorporated into RNA and DNA; traps and depletes DNA methyltransferases, reactivating silenced tumour-suppressor genes and inducing differentiation and apoptosis.
1.Azacitidine enters the cell and is phosphorylated
Source: US prescribing information (DailyMed). Doses are for orientation; the current label governs.
Injectable azacitidine (Vidaza and generics) is a Part B drug given subcutaneously or IV in the clinic. Oral azacitidine (Onureg) has a different indication (AML maintenance) and is a Part D drug.
Injectable generic azacitidine is covered on label without much review; Onureg requires prior authorisation and specialty pharmacy dispensing.
Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.
Sources: Medicare.gov: Chemotherapy · Medicare.gov: Prescription drugs (outpatient, Part B) · Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
Sources: NICE TA218 · SMC advice: azacitidine. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
First hypomethylating agent approved (MDS)
Onureg maintenance in AML
Venetoclax + azacitidine full approval in unfit AML (VIALE-A)
| Region | Year | Indication |
|---|---|---|
| US | 2004 | Myelodysplastic syndromes |
| US | 2020 | Newly diagnosed AML unfit for intensive chemotherapy, with venetoclax (VIALE-A) |
| US | 2020 | Oral azacitidine (Onureg) maintenance after intensive induction (QUAZAR AML-001) |
| Adverse event | Any grade | Grade 3+ |
|---|---|---|
| Neutropenia VIALE-A combination arm | - | 42% |
| Febrile neutropenia VIALE-A combination arm | - | 42% |
| Nausea | 44% | - |
| Injection-site reactions subcutaneous route | 30% | - |
Rates read from the US prescribing information. Blank cells mean the figure was not sourced, not that it is zero.
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AGILE showed that for the roughly 6-10% of AML patients with an IDH1 mutation, a targeted doublet produces survival in the range of two years, an outcome previously unimaginable in unfit patients. Ivosidenib-azacitidine is approved and is one option alongside venetoclax-azacitidine for these patients. Which regimen, or triplet, is best for IDH1-mutated disease has not been settled by a randomised trial.
The first maintenance therapy shown to lengthen survival in acute myeloid leukaemia; oral azacitidine (Onureg) was approved in the United States in 2020 for patients in first remission who cannot complete intensive curative therapy.
VIALE-A turned a palliative regimen into one that produces remission in two-thirds of older AML patients and is now the reference treatment for anyone not fit for intensive chemotherapy. It shifted the field towards lower-intensity targeted combinations and opened the door to adding FLT3, IDH and menin inhibitors to the backbone. Cure remains uncommon and most patients relapse within two years.
Azacitidine became the standard low-intensity treatment for older or unfit patients with AML and the control arm for later trials; venetoclax plus azacitidine (VIALE-A) now supersedes azacitidine alone where available.
Azacitidine (and decitabine) became the standard for higher-risk MDS in patients not going straight to transplant, and the backbone for combination trials that have so far failed to beat it.
Query for this drug: (TITLE:"Azacitidine" OR ABSTRACT:"Azacitidine" OR TITLE:"Vidaza" OR ABSTRACT:"Vidaza" OR TITLE:"Onureg" OR ABSTRACT:"Onureg" OR TITLE:"oral" OR ABSTRACT:"oral") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Azacitidine, not a curated reading list.
Shares Study of MP0533 in Patients Acute Myeloid Leukemia or Myelodysplastic Syndrome, Aptose Biosciences, A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1), Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tuspetinib (HM43239) in Patients With Relapsed or Refractory Acute Myeloid Leukemia.
Shares A Study of BGB-11417 in Participants With Myeloid Malignancies, A Phase 2 Study of MAX-40279 Combined With Venetoclax and Azacitidine in Unfit Newly Diagnosed Acute Myeloid Leukemia, VERONA, Study of SLS009 (Formerly GFH009) a Potent Highly Selective CDK9 Inhibitor in Patients With Hematologic Malignancies and High-Risk Newly Diagnosed AML.
Shares Venetoclax + hypomethylating agent, Shorter venetoclax courses in unfit AML, Unmask hidden antigens with a short epigenetic course before immunotherapy, Decitabine.
Shares Menin inhibitor + venetoclax + azacitidine, Menin / KMT2A (HOXA9-MEIS1 axis), Acute myeloid leukaemia (KEGG map), Epigenetic progenitor theory: cancer without a first mutation.
Shares An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy., AGILE, AGILE: ivosidenib plus azacitidine for newly diagnosed IDH1-mutated AML in patients unfit for intensive chemotherapy, Beat AML Master Trial.
Shares A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1), A Study of Gilteritinib, Venetoclax and Azacitidine as a Combined Treatment for People Newly Diagnosed With Acute Myeloid Leukemia, 7+3 induction chemotherapy, Beat AML Master Trial.
Shares A Study Evaluating Intensive Chemotherapy With or Without Glasdegib or Azacitidine With or Without Glasdegib In Patients With Previously Untreated Acute Myeloid Leukemia, Galinpepimut-S Versus Investigator's Choice of Best Available Therapy for Maintenance in AML CR2/CRp2, Study of Magrolimab in Combination With Azacitidine Versus Physician's Choice of Venetoclax in Combination With Azacitidine or Intensive Chemotherapy in Patients With TP53 Mutant Acute Myeloid Leukemia That Have Not Been Treated, A Study of BL-M11D1 in Combination With Cytarabine + Daunorubicin or Venetoclax + Azacitidine in Patients With Acute Myeloid Leukemia.
Shares Magrolimab + Azacitidine Versus Azacitidine + Placebo in Untreated Participants With Myelodysplastic Syndrome (MDS), Study of Magrolimab Versus Placebo in Combination With Venetoclax and Azacitidine in Participants With Acute Myeloid Leukemia, Study of Magrolimab in Combination With Azacitidine Versus Physician's Choice of Venetoclax in Combination With Azacitidine or Intensive Chemotherapy in Patients With TP53 Mutant Acute Myeloid Leukemia That Have Not Been Treated, Secondary and therapy-related acute myeloid leukaemia.